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BioMarin Inc elosulfase alfa, vimizim
Elosulfase Alfa, Vimizim, supplied by BioMarin Inc, used in various techniques. Bioz Stars score: 90/100, based on 1 PubMed citations. ZERO BIAS - scores, article reviews, protocol conditions and more
https://www.bioz.com/product/vimizim/elosulfase+alfa+vimizim/us12146169-43-9-10
Average 90 stars, based on 1 article reviews
elosulfase alfa, vimizim - by Bioz Stars, 2026-10
90/100 stars

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Article Title: Approvals, Submission, and Important Labeling Changes for US Marketed Pharmaceuticals
Article Snippet: Elosulfase alfa , Vimizim (BioMarin Pharmaceutical) , Treatment of patients suffering from mucopolysaccharidosis type IVA (MPS IVA) or morquio A syndrome. Priority review, but the date on which the agency will announce drug approval status was delayed by 3 months at the end of May 2013..

Article Title: Enzyme Replacement Therapies and Immunogenicity in Lysosomal Storage Diseases: Is There a Pattern?
Article Snippet: Based on findings from the Phase III trial, elosulfase alfa has an acceptable safety profile, with the most frequent adverse events being rated as mild to moderate infusion-associated reactions.12 In MOR-004 (the pivotal Phase III study), hypersensitivity reactions occurred in about one fifth of patients and were usually mild to moderate,12 but only 10% of patients in this trial tested positive for immunoglobulin E.13 Earlier multicenter, open-label dose-escalation trials (MOR-002) showed similar improvements in the 6MWT, which were sustained during almost 3 years of follow-up in the MOR-100 extension study.14 This maintenance of endurance over a period of years is clinically relevant for a patient population that, without treatment, would experience decline.9,15 *VIMIZIM, BioMarin Pharmaceutical Inc. (Novato, California).

Article Title: Ophthalmological Findings in Mucopolysaccharidoses
Article Snippet: Currently, there are four available ERTs—Aldurazyme ® (BioMarin Pharmaceutical Inc., Novato, CA, USA) for MPS I, Elaprase ® (Shire Human Genetic Therapies Inc., Cambridge, CA, USA) for MPS II, Vimizim ® (BioMarin Pharmaceutical Inc., Novato, CA, USA ) for MPS IVA, and Naglazyme ® (BioMarin Pharmaceutical Inc., Novato, CA, USA) for MPS VI [ ].

Article Title: Long-term effects of enzyme replacement therapy for Taiwanese patients with mucopolysaccharidosis IVA.
Article Snippet: Lin et al + MODEL alfa (Vimizim ; BioMarin Pharmaceutical Inc., Novato, CA, USA) is a recombinant human GALNS associated with increased endurance, reduced urine KS and acceptable safety profile, and it has been approved as enzyme replacement therapy (ERT) for MPS IVA.4,5 However, information regarding the long-term outcomes of ERT with elosulfase alfa for Taiwanese patients with MPS IVA is limited.

Recombinant:

Article Title: Fresh from the biotech pipeline-2014.
Article Snippet: 125 The US Food and Drug Administration (FDA) approved 41 new molecular entities (NME) in 2014, up substantially from last year’s approval total and surpassing the modern-era peak of 39 seen in 2012 (Fig. 1).. Biologics rebounded over last year’s slump, with a record 12 novel biologics license applications (BLAs) approved in 2014 (Table 1), including Thousand Oaks, California–based Amgen’s first-in-class bispecific antibody Blincyto (blinatumomab) and promising PD-1 (programmed death receptor 1) checkpoint inhibitors from Whitehouse Station, New Jersey–based Merck and Princeton, New Jersey–based Bristol-Myers Squibb (BMS).. It was also a banner year for drugs that treat rare diseases, which accounted for about 40% of all FDA nods.

Coagulation:

Article Title: Fresh from the biotech pipeline-2014.
Article Snippet: 125 The US Food and Drug Administration (FDA) approved 41 new molecular entities (NME) in 2014, up substantially from last year’s approval total and surpassing the modern-era peak of 39 seen in 2012 (Fig. 1).. Biologics rebounded over last year’s slump, with a record 12 novel biologics license applications (BLAs) approved in 2014 (Table 1), including Thousand Oaks, California–based Amgen’s first-in-class bispecific antibody Blincyto (blinatumomab) and promising PD-1 (programmed death receptor 1) checkpoint inhibitors from Whitehouse Station, New Jersey–based Merck and Princeton, New Jersey–based Bristol-Myers Squibb (BMS).. It was also a banner year for drugs that treat rare diseases, which accounted for about 40% of all FDA nods.

Produced:

Article Title: Fresh from the biotech pipeline-2014.
Article Snippet: 125 The US Food and Drug Administration (FDA) approved 41 new molecular entities (NME) in 2014, up substantially from last year’s approval total and surpassing the modern-era peak of 39 seen in 2012 (Fig. 1).. Biologics rebounded over last year’s slump, with a record 12 novel biologics license applications (BLAs) approved in 2014 (Table 1), including Thousand Oaks, California–based Amgen’s first-in-class bispecific antibody Blincyto (blinatumomab) and promising PD-1 (programmed death receptor 1) checkpoint inhibitors from Whitehouse Station, New Jersey–based Merck and Princeton, New Jersey–based Bristol-Myers Squibb (BMS).. It was also a banner year for drugs that treat rare diseases, which accounted for about 40% of all FDA nods.

Control:

Article Title: Fresh from the biotech pipeline-2014.
Article Snippet: 125 The US Food and Drug Administration (FDA) approved 41 new molecular entities (NME) in 2014, up substantially from last year’s approval total and surpassing the modern-era peak of 39 seen in 2012 (Fig. 1).. Biologics rebounded over last year’s slump, with a record 12 novel biologics license applications (BLAs) approved in 2014 (Table 1), including Thousand Oaks, California–based Amgen’s first-in-class bispecific antibody Blincyto (blinatumomab) and promising PD-1 (programmed death receptor 1) checkpoint inhibitors from Whitehouse Station, New Jersey–based Merck and Princeton, New Jersey–based Bristol-Myers Squibb (BMS).. It was also a banner year for drugs that treat rare diseases, which accounted for about 40% of all FDA nods.

Modification:

Article Title: Fresh from the biotech pipeline-2014.
Article Snippet: 125 The US Food and Drug Administration (FDA) approved 41 new molecular entities (NME) in 2014, up substantially from last year’s approval total and surpassing the modern-era peak of 39 seen in 2012 (Fig. 1).. Biologics rebounded over last year’s slump, with a record 12 novel biologics license applications (BLAs) approved in 2014 (Table 1), including Thousand Oaks, California–based Amgen’s first-in-class bispecific antibody Blincyto (blinatumomab) and promising PD-1 (programmed death receptor 1) checkpoint inhibitors from Whitehouse Station, New Jersey–based Merck and Princeton, New Jersey–based Bristol-Myers Squibb (BMS).. It was also a banner year for drugs that treat rare diseases, which accounted for about 40% of all FDA nods.

Clinical Proteomics:

Article Title: Current state of the art in treatment of Mendelian disease: Skeletal dysplasias.
Article Snippet: Funding information Greenberg Center for Skeletal Dysplasias Abstract The current state of the art in treatment of Mendelian disease, specifically skeletal dysplasias, benefits tremendously from Dr. Victor McKusick's early delineation and standardization of the nomenclature surrounding these conditions.. Through close observation and careful description of each dysplasia to flesh out the nosologic backbone of the genetic skeletal disorders, individuals with the same diagnosis were identified and grouped together for genetic interrogation.. These efforts have resulted in the identification of the genetic etiology of nearly all recognized skeletal disorders.



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